FORT LEE, N.J. – Kedrion Biopharma Inc., an international biopharmaceutical company specializing in the research and development, production, and commercialization of plasma-derived therapeutic products used in treating rare and serious diseases, announced today that plasminogen deficiency type 1 (PLGD-1) patients in the U.S. now have access to RYPLAZIM® (plasminogen, human-tvmh)....
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FORT LEE, N.J. — Kedrion Biopharma announced today that it has received FDA approval for QIVIGY immune globulin intravenous (human)-kthm 10% liquid, indicated for treatment of adults with primary humoral immunodeficiency (PI)—a serious and often underdiagnosed group of disorders that compromise the immune system’s ability to function effectively. QIVIGY, a 10% IVIG...
FORT LEE, N.J. — Kedrion is proud to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to investigational treatment for Congenital Aceruloplasminemia, a rare genetic disorder affecting iron metabolism. This designation marks a significant milestone in Kedrion’s commitment to addressing the unmet medical needs of patients living with ultra-rare...
A Keele researcher will embark on a two-year study to identify affordable treatments to help children living with spinal muscular atrophy. Dr Melissa Bowerman, of Keele University’s School of Medicine, has been awarded £99,959 by the Academy of Medical Science’s Springboard grant scheme to continue her research into treatments for...
PEA RIDGE – Kyler Kasper is living on borrowed blood. He has not yet celebrated his second birthday, but Kyler’s parents expect him to reach it, thanks to blood donations from strangers. Kyler was recently diagnosed with Diamond Blackfan anemia, a rare blood disorder that affects about 500 people in...
Brussels, Belgium – Patients are central to the rare disease community on all levels. This sentiment was echoed by representatives from all stakeholder groups at the 2023 World Orphan Drugs Congress which took place in early November 2023 in Barcelona. The Parliament joined the Alexion – AstraZeneca Rare Disease team...
BALTIMORE, Maryland — Researchers at Kennedy Krieger Institute are sharing their expertise on autism spectrum disorder in a medical journal reaching thousands of pediatric professionals worldwide. The journal, Pediatric Clinics, provides the latest clinical information on health and related issues for children and adolescents. The newly released volume is titled...
LEXINGTON, Mass. — Keros Therapeutics, Inc. (Keros) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta (TGF-ß) family of proteins, today announced the U.S. Food and...
LEXINGTON, Mass. — Keros Therapeutics, Inc. (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta (TGF-ß) family of proteins, today announced that the U.S. Food and...
NEW YORK — Keryx Biopharmaceuticals, Inc. (Nasdaq: KERX) today announced data on the clinical activity of KRX-0401 (perifosine), the Company’s Akt-inhibitor for cancer, as a treatment for advanced renal cell carcinoma. Abstract #5034, entitled, “Phase II study of perifosine in metastatic renal cell carcinoma (RCC) progressing after prior therapy (Rx)...